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Transcript-Targeted Therapy Based on RNA Interference and Antisense Oligonucleotides: Current Applications and Novel Molecular Targets.

The development of novel target therapies based on the use of RNA interference (RNAi) and antisense oligonucleotides (ASOs) is growing in an exponential way, challenging the chance for the treatment of the genetic diseases and cancer by hitting selectively targeted RNA in a sequence-dependent manner. Multiple opportunities are taking shape, able to remove defective protein by silencing RNA (e.g., Inclisiran targets mRNA of protein PCSK9, permitting a longer half-life of LDL receptors in heterozygous familial hypercholesteremia), by arresting mRNA translation (i.e., Fomivirsen that binds to UL1

The Effects of Artificial Sweeteners on Intestinal Nutrient-Sensing Receptors: Dr. Jekyll or Mr. Hyde?

The consumption of artificial and low-calorie sweeteners (ASs, LCSs) is an important component of the Western diet. ASs play a role in the pathogenesis of metabolic syndrome, dysbiosis, inflammatory bowel diseases (IBDs), and various inflammatory conditions. Intestinal nutrient-sensing receptors act as a crosstalk between dietary components, the gut microbiota, and the regulation of immune, endocrinological, and neurological responses. This narrative review aimed to summarize the possible effects of ASs and LCSs on intestinal nutrient-sensing receptors and their related functions. Based on the

Beyond Numbers: Uses and Value of In-Trial Interview Data for Regulatory and Health Technology Assessment (HTA) Decision Making.

This research explores the growing importance of qualitative in-trial research (ITR) in regulatory and health technology assessment (HTA) decision making. Since 2020, the Food and Drug Administration (FDA) and European Medicines Agency (EMA) have emphasized incorporating patient experience data into drug development and approval. We reviewed regulatory guidance documents, drug approval records, and HTA reports from January 2017 to March 2025. This included labels and reviews for new drug applications from the FDA and EMA, the Patient-Focused Drug Development guidance series, guidance on rare d

CRISPR-Cas9 Therapeutics in Early Clinical Development: Delivery and Molecular Diagnostics.

CRISPR-Cas9 has progressed from an experimental tool to a therapeutic modality, marked by the first regulatory approvals of an ex vivo-edited autologous CD34+ hematopoietic stem cell product that induces fetal hemoglobin (CASGEVY/exa-cel). In this narrative review, we synthesize modality-specific molecular diagnostic strategies used across early CRISPR clinical translation. In parallel, early clinical experience has begun to demonstrate the feasibility of in vivo editing, including subretinal delivery for CEP290-associated inherited retinal degeneration (EDIT-101 programme) and hepatocyte-targ

The role of heat shock protein 90 in idiopathic pulmonary fibrosis: state of the art.

Heat shock protein 90 (HSP 90) and its isoforms are a group of homodimeric proteins that regulate several cellular processes, such as the elimination of misfolded proteins, cell development and post-translational modifications of kinase proteins and receptors. Due to its involvement in extracellular matrix (ECM) remodelling, myofibroblast differentiation and apoptosis, HSP 90 has been investigated as a key player in the pathogenesis of lung fibrosis. Idiopathic pulmonary fibrosis (IPF) is the most common and deadly interstitial lung disease, due to the progressive distortion of lung parenchyma

Сфингосин-1-Фосфатные модуляторы для лечения ультразвукового колита: обзор наррации, сосредоточенный на безопасности.

Ульцеративный колит представляет собой хроническое иммуносодержащее воспалительное заболевание толстой кишки, которое вызывает значительную заболеваемость и повышает риск рака цвета. Был разработан ряд целевых методов лечения умеренно-сильного язвительного колита, что значительно улучшило его лечение. Озанимод и этирасимод, пероральные небольшие молекулярные препараты, являются последним дополнением. Они относятся к классу модуляторов сфингосин-1-фосфатного рецептора (S1PR) и являются первыми членами этого класса, получившими официальное утверждение на наличие язвительного колита. Они действую

Primary central nervous system lymphomas: EHA-ESMO Clinical Practice Guideline for diagnosis, treatment and follow-up.

This EHA-ESMO Clinical Practice Guideline provides key recommendations for managing primary DLBCL of the CNS.The guideline covers clinical, imaging and pathological diagnosis, staging and risk assessment, treatment and follow-up.Algorithms for first-line and salvage treatments are provided.The author group encompasses a multidisciplinary group of experts from different institutions and countries in Europe.Recommendations are based on available scientific data and the authors' collective expert opinion.

The Clinical Application of Ozonetherapy

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Pericytes as targets in hereditary hemorrhagic telangiectasia.

Defective paracrine Transforming Growth Factor-β (TGF-β) signaling between endothelial cells and the neighboring mural cells have been thought to lead to the development of vascular lesions that are characteristic of Hereditary Hemorrhagic Telangiectasia (HHT). This review highlights recent progress in our understanding of TGF-β signaling in mural cell recruitment and vessel stabilization and how perturbed TGF-β signaling might contribute to defective endothelial-mural cell interaction affecting vessel functionalities. Our recent findings have provided exciting insights into the role of thalid

Конвергенция глобальных эпидемий: диабет как модулятор нейродегенеративных и нейро-воспалительных расстройств.

Диабет меллитус (ДМ) и неврологические расстройства быстро совпадают с глобальным бременем на здоровье, обусловленным старением населения, растущей распространенностью метаболического синдрома, а также ограниченностью возможностей раннего обнаружения и коррекции заболеваний для многих неврологических синдромов. Помимо своей устоявшейся роли в периферической нейропатии, связанной с диабетом, DM все чаще используется в качестве изменяющего фактора риска, фенотипа и прогнозирования широкого спектра заболеваний центральной и периферической нервной системы. В ходе этого обзора мы обобщаем текущие э

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… They may reduce autoimmune β -cell injury (relevant to T1D) ( 97 ), improve insulin resistance and metabolic inflammation (T2D), attenuate diabetic nephropathy via S1P1-dependent endothelial and anti-fibrotic pathways ( 98 ), and stabilize the blood-retinal barrier in early diabetic retinopathy ( 99 …

Bilateral Proptosis in a Case of Recurring Multiple Myeloma: Uncommon Orbital Presentation of Plasmacytoma.

Multiple myeloma is a malignant plasma cell dyscrasia that may invade the orbits in extramedullary presentations. Common manifestations of orbital involvement include unilateral proptosis, injection, chemosis, diplopia, and occasionally pain. Additionally, the soft-tissue tumors associated with multiple myeloma typically cause bony destruction of adjacent structures. However, in certain patients, bilateral proptosis and a lack of bony destruction may occur. In these instances, it is important for physicians to consider patient history, presentation, and progression in order to ensure that a po

The Potential of Chronotherapy and Nanotherapy-Based Strategies for Glioblastoma Treatment.

Glioblastoma is the most common and aggressive brain tumour in adults, and despite ongoing efforts, effective treatment remains limited. Standard therapies often face challenges because of the tumour's specific biology, its aggressive nature, and the presence of certain physiological barriers in the brain that impede chemotherapeutics from reaching their target. Emerging research in circadian biology highlights the role of the internal circadian clock in tumour progression and treatment response. Evidence suggests that aligning therapy to patients' chronotypes could potentially improve treatme

Диагностическая полезность индекса свободной цепи света в Каппе у взрослых с оптовым неритом.

Простая, быстрая и воспроизводимая процедура различения множественного склероза (MS), миелин олигодендроцита гликпротеина, связанного с антителами (MOGAD), и спектрального расстройства нейромиелита (NMOSD) при первом оптическом неуриите (ION) может быть весьма полезной для руководства ранним управлением. Мы включили всех взрослых, принятых в центр MS в Марселе для ION в период с марта 2016 и апрель 2024, с анализом CSF, включая индекс свободной цепи освещения (K-FLC). Для измерения диагностической способности индекса К-ФЛК использовались кривые эксплуатационных характеристик получателя.Результ

The role and mechanisms of mesenchymal stem cells in postoperative cognitive dysfunction via immune network regulation.

Postoperative cognitive dysfunction (POCD) is a common neurological complication in elderly patients after surgery. Its pathophysiology is complex. Neuroinflammation, oxidative stress, and immune imbalance are all involved in this process. These changes can affect postoperative recovery and reduce patients' quality of life. As population aging continues to increase, the prevention and treatment of POCD have become important clinical issues. In recent years, mesenchymal stem cells (MSCs) have attracted attention in neurological diseases because of their immunomodulatory and tissue healing abili

Secondary Immunodeficiency in Hematological Malignancies: Focus on Multiple Myeloma and Chronic Lymphocytic Leukemia.

Secondary immunodeficiency is reported in most patients with hematological malignancies such as chronic lymphocytic leukemia and multiple myeloma. The aim of our review was to evaluate the existing literature data on patients with hematological malignancies, with regard to the effect of immunodeficiency on the outcome, the clinical and therapeutic approach, and on the onset of noninfectious complications, including thrombosis, pleural effusion, and orofacial complications. Immunodeficiency in these patients has an intense impact on their risk of infection, in turn increasing morbidity and mort

Разоблачение связи: изучение причинно-следственной связи между диабетом, множественным склерозом, мигренью и болезнью Альцгеймера посредством менделийской случайности.

ВВЕДЕНИЕ: Согласно результатам наблюдений, сахарный диабет [тип] 1 сахарный диабет (T1DM), тип 2 Заболевание диабетом (T2DM)], множественным склерозом (MS) и мигренью связано с болезнью Альцгеймера (AD). Однако причинно-следственная связь не была в полной мере выяснена. Таким образом, мы намерены оценить причинно-следственную связь между T1DM, T2DM, MS и мигренью с риском использования AD two- выборка менделианского исследования случайности (MR). МЕТОДЫ: Генетические инструменты были выявлены для AD, T1DM, T2DM, MS и мигрени, соответственно, на основе исследования, проведенного в рамках всей а

Advances in antisense oligonucleotide treatment for cancer.

RNA therapeutics, including antisense oligonucleotides (ASOs), have emerged as a promising class of drugs, with several already approved for clinical use. To date, most approved ASO-based RNA therapies target non-malignant disorders such as neurodegenerative diseases, and only a single therapy in this class has been approved for cancer. Notably, nearly half of existing RNA therapeutics act by modulating RNA splicing. Given the growing evidence implicating aberrant RNA splicing in cancer pathogenesis, the development of ASO-based therapeutics for oncologic indications is expected to accelerate.

RNA therapeutics in targeting G protein-coupled receptors: Recent advances and challenges.

G protein-coupled receptors (GPCRs) are the major targets of existing drugs for a plethora of human diseases and dominate the pharmaceutical market. However, over 50% of the GPCRs remain undruggable. To pursue a breakthrough and overcome this situation, there is significant clinical research for developing RNA-based drugs specifically targeting GPCRs, but none has been approved so far. RNA therapeutics represent a unique and promising approach to selectively targeting previously undruggable targets, including undruggable GPCRs. However, the development of RNA therapeutics faces significant cha

MicroRNA-7: a versatile player and core target in brain disorders.

OBJECTIVE: This article provides a systematic review of the biological characteristics, regulatory mechanisms, and roles of MicroRNA-7 (miR-7) in the nervous system, as well as explores its clinical translation potential and challenges. METHODS: A review analysis integrating the progress of basic and clinical research related to miR-7. RESULTS: Characteristics and Regulation: miR-7 is highly expressed in mammalian brain tissues, with its function finely regulated by ceRNA networks (such as ciRS-7 and lncRNA SNHG1) and epigenetic modifications, forming a multi-layer dynamic regulatory system.

Infectious complications after hematopoietic stem cell transplantation: current status and future perspectives in Korea.

Hematopoietic stem cell transplantation (HSCT) is a treatment for hematologic malignancies, immune deficiencies, or genetic diseases, ect. Recently, the number of HSCTs performed in Korea has increased and the outcomes have improved. However, infectious complications account for most of the morbidity and mortality after HSCT. Post-HSCT infectious complications are usually classified according to the time after HSCT: pre-engraftment, immediate post-engraftment, and late post-engraftment period. In addition, the types and risk factors of infectious complications differ according to the stem cell